Evidence map›Paper›PMID 29606505›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018

Prevention of Photoreceptor Cell Loss in a Cln6

Sophia-Martha Kleine Holthaus, Joana Ribeiro, Laura Abelleira-Hervas, Rachael A Pearson, Yanai Duran, Anastasios Georgiadis, Robert D Sampson, Matteo Rizzi, Justin Hoke, Ryea Maswood and 5 more

Open access · bronzeAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.

0numbers the graph read from it
0cells of the map it votes in
25citing papers in PubMed
4.4field-weighted citation impact, top 5% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

25 citing papers in PubMed, 45 citations in OpenAlex.

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  17. [Experimental therapeutic approaches for the treatment of retinal dystrophy in neuronal ceroid lipofuscinosis].Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft · 2021
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors at 2 institutions in 1 country.

Sophia-Martha Kleine HolthausDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK; MRC Laboratory for Molecular Cell Biology, University College London, Gower Street, London WC1E 6BT, UK.
Joana RibeiroDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Laura Abelleira-HervasDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Rachael A PearsonDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Yanai DuranDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Anastasios GeorgiadisDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Robert D SampsonDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Matteo RizziDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Justin HokeDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Ryea MaswoodDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Selina AzamDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Ulrich F O LuhmannDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Alexander J SmithDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Sara E MoleMRC Laboratory for Molecular Cell Biology, University College London, Gower Street, London WC1E 6BT, UK; UCL Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; UCL Department of Genetics, Evolution and Environment, University College London, Gower Street, London WC1E 6BT, UK. Electronic address: s.mole@ucl.ac.uk.
Robin R AliDepartment of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, City Road, London EC1V 2PD, UK. Electronic address: r.ali@ucl.ac.uk.
University College London · GBMRC Laboratory for Molecular Cell Biology · GB

Funding

Department of HealthMedical Research Council
6 · The paper itself

Abstract

The neuronal ceroid lipofuscinoses (NCLs) are inherited lysosomal storage disorders characterized by general neurodegeneration and premature death. Sight loss is also a major symptom in NCLs, severely affecting the quality of life of patients, but it is not targeted effectively by brain-directed therapies. Here we set out to explore the therapeutic potential of an ocular gene therapy to treat sight loss in NCL due to a deficiency in the transmembrane protein CLN6. We found that, although Cln6

Indexed as

AnimalsDependovirusDisease Models, AnimalGene ExpressionGenetic TherapyGenetic VectorsGene Transfer TechniquesHumansImmunohistochemistryMembrane ProteinsMiceMice, TransgenicNeuronal Ceroid-LipofuscinosesPhotoreceptor CellsRetinal Bipolar CellsCln6 protein, mouseMembrane ProteinsAAV gene therapyBatten diseasebipolar cellslysosomal storage disordersneuronal ceroid lipofuscinosesphotoreceptorsretina

Identifiers

PMID29606505
PMCPMC5993939
OpenAlexW2789820265

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.